Expanded Access

Expanded Access Opportunities

Active, No Longer Enrolling

RAPA-501 EAP, by Rapa Therapeutics

In people with ALS, the body’s immune system becomes imbalanced and appears to hasten the loss of motor neurons in the brain and spinal cord. Regulatory T-cells help reduce inflammation and could lead to a more balanced immune system in people with ALS. The goal of this study is to reduce neuroinflammation, potentially slowing ALS progression, using specially prepared regulatory T-cells, called RAPA-501 cells. RAPA-501 cells are created through a series of steps by first taking the participant’s own blood though a specialized IV (apheresis), then isolating regulatory T-cells from the blood. Next, these regulatory T-cells are grown under special conditions in a petri dish, becoming RAPA-501 cells. The RAPA-501 cells are then returned to the participant through an intravenous infusion. Prior to the IV infusion of RAPA-501 cells, a low dose of chemotherapy is given to reduce the body’s immune response and potentially heighten the effects of the RAPA-501 cells.

View the RAPA-501 EAP on ClinicalTrials.gov

 Download the RAPA-501 EAP Brochure

Watch a webinar about the RAPA-501 EAP

CNM-Au8.EAP04, by Clene Nanomedicine

Motor neurons consume significant energy in order to function normally. In ALS, corrupted energy metabolism together with increased cellular stress lead to motor neuron degeneration. CNM-Au8 is a new class of medicine that provides an energetic assist to impaired motor neurons, helping them improve their ability to function more normally. CNM-Au8 acts catalytically to both support bioenergetic reactions inside cells and eliminate the harmful waste byproducts of cellular metabolism. Oral delivery of CNM-Au8 resulted in both neuroprotection and remyelination in multiple animal studies. Each 2 oz dose of CNM-Au8 is a concentrated, liquid suspension of pure gold nanocrystals that study participants drink every morning. These extremely small nanocrystals travel through the body and enter the brain and motor neuron cells where they enhance the ability of these cells to survive and communicate by supporting cellular metabolism. CNM-Au8 was demonstrated to be safe and well-tolerated by healthy volunteers in a Phase 1 study.

View the CNM-Au8.EAP04 on ClinicalTrials.gov

Pridopidine EAP2, by Prilenia Therapeutics

Pridopidine is a highly selective Sigma-1 receptor (S1R) agonist developed by Prilenia for the treatment of neurodegenerative and neurodevelopmental disorders. S1R regulates key cellular pathways, commonly impaired in neurodegeneration. Of particular interest is its role in the pathogenesis of ALS which is supported by human genetic and postmortem studies as well as by preclinical models. Pridopidine demonstrates robust neuroprotective effects in numerous preclinical models of neurodegenerative diseases including models of ALS. Compelling preclinical data supports the therapeutic potential of pridopidine in ALS. In ALS SOD1G93A motor neurons (MNs), pridopidine exerts neuroprotective effects via activation of the S1R. Specifically, pridopidine increases MN survival, improves BDNF and GDNF axonal transport, and restores the neuro-muscular junction (NMJ) synaptic activity. In vivo, pridopidine treatment of SOD1G93A mice reduces toxic protein aggregates and ameliorates muscle fiber wasting.

View the Pridopidine EAP on ClinicalTrials.gov

Download the Pridopidine EAP Brochure

Watch a webinar about the Pridopidine EAP

Common Questions About Expanded Access

The best way to learn about expanded access opportunities is to have a discussion with your ALS doctor and clinical care team. Below are some common questions and resources that can help guide these discussions.

If you have additional questions, please register for our community webinars. These webinars are an excellent opportunity to access reliable information from leading ALS researchers, clinicians, and industry professionals.

How is expanded access different from clinical trials?

The purpose of expanded access is to provide access to an investigational product (IP). Expanded Access Protocols (EAPs) should not interfere with the completion of clinical trials; therefore, EAPs are meant to be an option for patients who do not qualify for clinical trials. The criteria for participation in an EAP are broad, in-person visits may be infrequent, and there is no placebo. Expanded access is considered an extension of clinical care. Visit ClinicalTrials.govto explore available EAP opportunities.

On the other hand, the purpose of clinical trials is to formally test investigational products (IPs). Data gathered from clinical trials may lead to FDA approval of new treatments if an IP is found to be safe and effective. Clinical trials have specific eligibility criteria, frequent in-person study visits, and typically involve a placebo group. Clinical trials are separate from clinical care. View clinical trial opportunities at the Healey & AMG Center for ALS.

What is an EAP?

EAP stands for Expanded Access Protocol, which is an opportunity for people with life-threatening conditions or serious diseases who are not eligible for clinical trials to gain access to an investigational product that has been shown to be safe and tolerable in early clinical trials. Investigational products offered through EAPs are currently being studied in clinical trials and are not yet approved by the FDA. To read more about expanded access, visit the FDA website.

Am I eligible to participate in EAPs?

Eligibility criteria will differ from one EAP to another depending on the unique properties of the investigational product being offered. To find out if you may be eligible to participate in an EAP, please talk to your ALS doctor or contact a research center that is offering an EAP of interest to you. Use the search tool on ClinicalTrials.gov to explore available EAP opportunities.

Why would I participate in an EAP?

Expanded Access Protocols (EAPs) give people living with ALS who are not eligible for clinical trials the opportunity to take an investigational product (IP) while the IP is being formally tested. Participation in an EAP may also contribute to ALS research by providing safety and biomarker data. If you are interested in participating in an EAP, it is helpful to have a conversation with your ALS doctor to discuss the potential risks and benefits.

How are EAPs funded?

Expanded Access Protocols (EAPs) are typically funded by multiple sources, including contributions from grants, industry partners, philanthropy, and fundraising. The costs associated with an EAP include the supply, administration, and storage of the investigational product. Funding also supports the staff (clinicians, nurses, and research coordinators) who oversee clinical monitoring, in-person appointments, and virtual visits for each EAP participant. For specific information about patient care costs associated with an EAP, please talk to your clinical care team.

How can I learn more about EAPs?

The best way to learn about Expanded Access Protocol (EAP) opportunities is to have a discussion with your ALS doctor and clinical care team. Your care team may be offering an EAP or may be able to provide you with information about multicenter EAPs near you.

It may be possible for a licensed medical doctor to approach a pharmaceutical company to request an EAP on behalf of his/her patient. FDA approval and factors such as financial and staffing resources may determine whether a doctor can offer an EAP at your care center. Additionally, a pharmaceutical company must be able to supply the investigational product (IP) requested for an EAP.